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A new drug called osimertinib could improve survival in non-small cell lung cancer, a new clinical trial confirms. Image credit: Xvision/Getty Images.
  • Non-small cell lung cancer is the most common type of lung cancer.
  • A clinical trial has found that osimertinib, which is a targeted therapy for this type of cancer, improved patients’ chances of survival after surgery.
  • Osimertinib was better than placebo at reducing the risk of the cancer spreading to the central nervous system, and appears to have outperformed older drugs in the same class tested in other trials.
  • The trial confirmed that the drug is safe and well tolerated by patients.

According to the American Cancer Society, 80–85% of all lung cancers are a type known as non-small cell lung cancer (NSCLC).

The biggest risk factor for this kind of lung cancer is smoking. Some patients experience no symptoms in the early stages of the disease, but of those who do develop symptoms, a common first sign is an unexplained cough that will not go away.

After diagnosis, surgeons can remove part or all of the affected lung, known as “surgical resection.”

Chemotherapy can then prevent the cancer from coming back after surgery. However, the risk of recurrence increases in step with how advanced the initial cancer was.

In patients with NSCLC, the cancer often spreads, or metastasizes, into other parts of the body, especially the central nervous system (CNS).

A class of drugs known as epidermal growth factor receptor inhibitors can improve patients’ chances of disease-free survival. The drugs block a receptor that promotes uncontrolled cell division, which is the hallmark of cancer.

Results from a clinical trial now show that a new inhibitor in this class, called osimertinib, is safe and improves patients’ chances of disease-free survival after surgery.

The researchers speculate that the drug may also be more effective than existing inhibitors at preventing the cancer from spreading to the CNS.

“This therapy was well tolerated and prevented patients from developing metastasis to distant sites such as the brain, bone, and other areas of the lungs,” says lead investigator Dr. Roy S. Herbst, ensign professor of medicine (medical oncology), professor of pharmacology, and deputy director at Yale Cancer Center in New Haven, CT.

“This has truly impacted the lives of our patients,” he adds.

The results of the clinical trial appear in the Journal of Clinical Oncology.

The trial recruited 682 patients with NSCLC who had undergone a complete lung resection, with or without subsequent chemotherapy.

All of the patients tested positive for a mutation in the gene that manufactures epidermal growth factor receptor (EGFR). The mutation increases the activity of this receptor, which osimertinib is designed to block.

“EGFR mutations are the most common mutation identified in people with lung cancer who have never smoked,” Dr. Nadia Yousaf, a consultant medical oncologist at The Royal Marsden NHS Foundation Trust in London, United Kingdom, who was not involved in the study, explained for Medical News Today.

“All lung cancers diagnosed in the U.K. are routinely screened for this mutation,” she added.

Dr. William Dahut, chief scientific officer at the American Cancer Society, noted that this is also the case in the United States.

“The standard of care [in the U.S.] is for all non-small cell lung cancer patients to undergo molecular analysis of their tumors to look for potential targeted therapies, including EGFR,” he told us, adding, however, that “[s]adly this is not always done.”

In the clinical trial, the researchers randomly assigned patients to take either 80 milligrams of osimertinib or a placebo pill once a day for up to three years.

After 4 years, 73% of the patients who took osimertinib were alive and cancer-free, compared with 38% of those who took the placebo.

Fewer patients in the osimertinib group developed metastases locally or elsewhere in the body, including in the CNS, compared with patients in the placebo group.

In their paper, the researchers say osimertinib may be better than older drugs in the same class at preventing the spread of cancer to the CNS.

However, they note that clinical trials of these other drugs involved different patient populations and follow-up times. This makes it difficult to make direct comparisons between their results and the new trial.

The newly published study is an update of a previous paper, which reported results after 2 years of treatment with osimertinib.

The authors conclude:

“These updated data highlight the importance of routine EGFR testing at diagnosis to ensure that patients have the opportunity for optimal treatment.”